Rare Daily Staff

The European Commission’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion on Vertex Pharmaceuticals cystic fibrosis drug Kalydeco for children aged 12 to 24 months.

Cystic fibrosis (CF) is a rare, life-shortening genetic disease. It is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

Kalydeco is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, the drug is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

The positive opinion for Kalydeco would expand the approved use of the drug to include the treatment of people with CF aged 12 to <24 months who have at least one of nine mutations in their CFTR gene. If the European Commission issues a favorable adoption of the CHMP opinion for the extension of indication, Kalydeco will be the first and only medicine approved in Europe to treat the underlying cause of CF for these patients.

“Cystic fibrosis is a chronic, progressive disease that is present at birth, with symptoms often occurring in infancy, so early treatment is crucial to deliver the best possible outcomes for patients,” said Reshma Kewalramani, executive vice president, global medicines development and medical affairs and chief medical officer at Vertex. “Today’s announcement marks an important step towards allowing young CF patients to benefit from treatment at an early stage of their disease, and brings us one step closer to our goal of treating all people living with CF.”

 

October 22, 2018
Photo: Reshma Kewalramani, executive vice president, global medicines development and medical affairs and chief medical officer at Vertex